Biogen Idec

The experimental antisense drug will be tested in infants with a genetic spinal muscular atrophy diagnosis who are to date without symptoms

posted on March 16, 2015 - 11:03am

Biogen Idec has announced plans to run a phase 2 clinical study of the experimental drug ISIS-SMNRx in infants with spinal muscular atrophy (SMA)who are less than 6 weeks old and have a genetic diagnosis of the disorder but are not yet showing symptoms.

Isis Pharmaceuticals has announced the opening of a U.S. trial to evaluate the safety and tolerability of ISIS-DMPKRx in adults with type 1 myotonic dystrophy

posted on December 22, 2014 - 5:12pm

A phase 1-2 trial of ISIS-DMPKRx, an experimental compound for the treatment of type 1 myotonic muscular dystrophy, is now underway in the U.S. in adults with this disorder who are 20 to 55 years old and meet other study criteria.

Isis Pharmaceuticals has opened a second phase 3 trial to test its "antisense" drug for spinal muscular atrophy in children ages 2 to 12

posted on November 21, 2014 - 10:19am

Update (Dec. 1, 2014): Isis has announced that its partner, Biogen Idec, plans to conduct a phase 2 trial of ISIS-SMNRx in up to 25 presymptomatic newborns genetically predisposed to develop SMA, as well as a phase 2 study of this drug in approximately 20 patients with infantile or childhood-onset SMA who do not meet the inclusion criteria for the current phase 3 studies. These two additional...

Isis Pharmaceuticals has launched a trial to test multiple doses of its experimental antisense drug ISIS-SMNRx in 24 children with SMA at four US centers

posted on November 15, 2012 - 2:45pm

Update (Nov. 22, 2013): In a Nov. 22, 2013, press release, Isis Pharmaceuticals announced it would investigate a higher dose of ISIS-SMNRx in children with SMA than originally planned. It will add a 12-milligram cohort to the ongoing phase 1b/2a study.